AIRNA Announces First Patient Dosed in Phase 1 Trial of AIR-001, Potential Best-in-Class RNA-Editing Therapy for Alpha-1 Antitrypsin Deficiency (AATD)

- U.S. Food and Drug Administration grants Orphan Drug Designation to AIR-001 for treatment of AATD -

- Clearance received from regulatory authorities in multiple countries to initiate Phase 1 clinical study -

- Global study will expand to enroll patients at an estimated 20 sites across 11 countries -

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AIRNA, a biotech company pioneering RNA-editing therapeutics to transform the lives of patients with rare and common conditions, today announced the first patient has been dosed in the global Phase 1 clinical trial of AIR-001, AIRNA’s potential best-in-class RNA-editing candidate for the treatment of alpha-1 antitrypsin deficiency (AATD).

“RNA editing has the potential to deliver functional cures to people living with inherited diseases like AATD,” said Jacob S. Elkins, M.D., Chief Medical Officer of AIRNA. “Promising preclinical data indicate AIR-001 has the potential to increase functional AAT levels to address both the lung and liver manifestations of AATD, with a reversible and repeatable dosing approach. We are excited to carry this momentum forward in our RepAIR1 global clinical study and begin translating these findings into meaningful outcomes for patients.”

The Company additionally announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to AIR-001 for the treatment of AATD.

AATD is a genetic disease caused by mutations in the SERPINA1 gene that reduce levels of functional alpha-1 antitrypsin (M-AAT) protein, leading to progressive lung disease and liver injury. AIR-001, a subcutaneously delivered GalNAc oligonucleotide, is designed to correct the most common disease-causing mutation of AATD (PiZ) at the RNA level by recruiting endogenous ADAR enzymes to precisely edit SERPINA1 mRNA. RNA editing works by correcting disease-causing mutations at the RNA level without cutting or permanently altering DNA, enabling a reversible and repeatable treatment approach. By repairing the underlying genetic defect and restoring AAT protein levels, AIR-001 has the potential to address both the lung and liver manifestations of AATD.

The Phase 1 clinical trial, called RepAIR1 (NCT07431112), is an open-label, integrated single ascending and multiple dose study in adults with AATD who have the PiZZ genotype. It is designed to evaluate the safety, PK, and PD response to AIR-001, as assessed by changes in total AAT, functional AAT, Z-AAT and M-AAT. It will enroll approximately 54 patients. The trial has received authorization from regulatory authorities in multiple countries. It is currently enrolling patients in Australia and the United Kingdom and is expected to eventually expand to an estimated 20 sites across 11 countries.

About AIR-001
AIR-001 is AIRNA’s lead RNA-editing therapeutic candidate for alpha-1 antitrypsin deficiency (AATD), a genetic disease most commonly caused by the PiZ mutation in the SERPINA1 gene. AIR-001 is designed to repair the underlying cause of disease and restore production of functional alpha-1 antitrypsin protein, with the goal of addressing both the lung and liver manifestations of AATD. Administered via subcutaneous injection, AIR-001 is designed for convenient, repeat dosing. AIR-001 has been granted Orphan Drug Designation for the treatment of alpha-1 antitrypsin deficiency from the U.S. Food and Drug Administration.

About AIRNA
AIRNA is a clinical-stage company developing RNA-editing medicines designed to repair disease-causing genetic variants and introduce beneficial variants that promote human health. The company’s proprietary platform is based on research by RNA-editing pioneers and co-founders Thorsten Stafforst (University of Tübingen) and Jin Billy Li (Stanford University). AIRNA is advancing a pipeline of RNA-editing therapeutics led by a product candidate for alpha-1 antitrypsin deficiency (AATD). AIRNA is headquartered in Cambridge, MA, with research operations in Tübingen, Germany. Learn more at https://airna.com/.